RN1201 Injection in Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma

NCT07114432 | Not Yet Recruiting Phase 1

• 1 other identifier: NDHR
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 2

Brief Summary

This is a single-arm, dose-escalation exploratory study evaluating the safety and efficacy of RN1201, a BCMA/CD19-targeted allogeneic CAR-T cell therapy, in patients with newly diagnosed cytogenetically high-risk multiple myeloma who are ineligible or unwilling to undergo autologous stem cell transplantation (ASCT). Patients will receive lymphodepletion followed by a single infusion of RN1201 across four dose levels. Primary endpoints include incidence and severity of treatment-emergent adverse events. Secondary endpoints assess response rate and minimal residual disease (MRD) status.

Conditions

Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT; Newly Diagnosed Multiple Myeloma (NDMM)

Keywords

Newly Diagnosed; High-Risk Cytogenetic; Multiple Myeloma

Outcome Measures

Primary Outcomes (2)

• The incidence and severity of dose-limiting toxicities (DLTs)

  DLTs: Within 28 days after CAR-T cell infusion

• The incidence and severity of treatment-emergent adverse events (TEAEs)

  TEAEs: From infusion up to 24 months post-treatment.

Secondary Outcomes (5)

• Overall effectiveness and duration of efficacy

  4 weeks, 3 months, 6 months, and 12 months

• Pharmacokinetic (PK) of RN1201

  From RN1201 infusion up to 12 months

• Levels of Peripheral blood M protein

  From RN1201 infusion up to 12 months

• Levels of urine M protein

  From RN1201 infusion up to 12 months

• Levels of Peripheral blood cytokine

  From RN1201 infusion up to 12 months

Study Arms (1)

Allogeneic CAR-T cell therapy

EXPERIMENTAL

Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT are treated with allogeneic CAR-T cell therapy

Drug: BCMA/CD19-targeted allogeneic CAR-T

Interventions

BCMA/CD19-targeted allogeneic CAR-T DRUG

Lymphodepletion chemotherapy followed by allogeneic CAR-T cell (RN1201) infusion

Also known as: RN1201

Allogeneic CAR-T cell therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• A subject will be eligible for this trial only if all of the following criteria are met:
• Is willing and able to provide a written informed consent form before any trial-related activities are performed.
• Must have a diagnosis of Multiple Myeloma (MM) according to the World Health Organization (WHO) 2017 revised criteria.
• Must be diagnosed with active MM according to the International Multiple Myeloma Working Group (IMWG) criteria.
• Must meet the definition of genetic high-risk MM as per the "2024 Chinese Expert Consensus on the Diagnosis and Treatment of High-Risk Multiple Myeloma", defined as having one or more of the following high-risk cytogenetic abnormalities (HRCA) confirmed by FISH or mitotic karyotype analysis:
• del(17p) TP53 mutation t(4;14) t(14;16) t(14;20) 1q21 gain/amplification (defined as copy number ≥ 4) Del 1p Note: 1q21 amplification alone does not define cytogenetic high risk.
• Must have a documented MM type that is B-cell maturation antigen (BCMA) positive and/or CD19 positive, confirmed at screening or from prior medical records.
• Age ≥ 18 years, male or female.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 at enrollment.
• Has a life expectancy of ≥ 12 weeks.
• Meets the following prior therapy requirements:
• Is currently receiving or has received an induction therapy regimen containing at least a proteasome inhibitor, an immunomodulatory agent, or a monoclonal antibody as a backbone therapy.
• Has not received more than 4 cycles of induction therapy at the time of enrollment.
• Has had an effective response to induction therapy without evidence of disease progression per IMWG criteria.
• Prior radiotherapy is permitted before enrollment.

You may not qualify if:

• A subject will not be eligible for this trial if any of the following criteria are met:
• Known history of allergic reaction, hypersensitivity, intolerance, or contraindication to the BCMA/CD19 allogeneic CAR-T product or any of its excipients, including fludarabine, cyclophosphamide, and tocilizumab.
• Diagnosis of plasma cell leukemia.
• Presence of non-paraskeletal extramedullary disease.
• Relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) with active graft-versus-host disease (GVHD) that requires treatment with steroids or immunosuppressive agents.
• Presence of a severe, active infection, including:
• Human Immunodeficiency Virus (HIV) infection (subjects must have a negative HIV 1/2 antibody screen).
• Hepatitis C Virus (HCV) infection (subjects must have a negative anti-HCV antibody screen).
• Hepatitis B Virus (HBV) infection (subjects must have a negative HBsAg screen).
• History of prior gene therapy or genetically modified cell immunotherapy.
• Active autoimmune disease, including connective tissue disease, uveitis, sarcoidosis, inflammatory bowel disease, or multiple sclerosis; or a history of a severe autoimmune disease (as judged by the Principal Investigator) that required long-term immunosuppressive therapy.
• Diagnosis of an acquired or congenital immunodeficiency disease.
• History of Class III or IV heart failure as defined by the New York Heart Association (NYHA), unstable angina, myocardial infarction within the last 6 months, or sustained (\>30 seconds) ventricular arrhythmia.
• History of seizure disorder or other central nervous system (CNS) disease; history or current evidence of CNS involvement with MM. Note: CNS screening (e.g., lumbar puncture) is not mandatory unless symptoms are present.
• History of other primary malignancies, except for the following:

Sponsors & Collaborators

• Tianjin Medical University Cancer Institute and Hospital: lead
• Allorunning Therapeutics: collaborator

Study Sites (2)

Tianjin Medical University Cancer Institute and Hospital

Tianjin, Tianjin Municipality, 300060, China

Location

Tianjin Cancer Hospital Airport Hospital

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Conditions

Multiple Myeloma

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Yafei Wang, PhD

  Tianjin Medical University Cancer Institute and Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yafei Wang, PhD

CONTACT

+86-18622221250; drwang2005@163.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2025
• First Posted: August 11, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): August 1, 2028
• Last Updated: August 11, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)